Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare

Background Advanced therapy medicinal products (ATMPs) are beginning to reach European markets, and questions are being asked about their value for patients and how healthcare systems should pay for them. Objectives To identify and discuss potential challenges of ATMPs in view of current health technology assessment (HTA) methodology—specifically economic evaluation methods—in Europe as it relates to ATMPs, and to suggest potential solutions to these challenges. Methods An Expert Panel reviewed current HTA principles and practices in relation to the specific characteristics of ATMPs. Results Three key topics were identified and prioritised for discussion—uncertainty, discounting, and health outcomes and value. The panel discussed that evidence challenges linked to increased uncertainty may be mitigated by collection of follow-on data, use of value of information analysis, and/or outcomes-based contracts. For discount rates, an international, multi-disciplinary forum should be established to consider the economic, social and ethical implications of the choice of rate. Finally, consideration of the feasibility of assessing the value of ATMPs beyond health gain may also be key for decision-making. Conclusions ATMPs face a challenge in demonstrating their value within current HTA frameworks. Consideration of current HTA principles and practices with regards to the specific characteristics of ATMPs and continued dialogue will be key to ensuring appropriate market access. Classification code I

The apparent roughness of a sand surface blown by wind from an analytical model of saltation

We present an analytical model of aeolian sand transport. The model quantifies the momentum transfer from the wind to the transported sand by providing expressions for the thickness of the saltation layer and the apparent surface roughness. These expressions are derived from basic physical principles and a small number of assumptions. The model further predicts the sand transport rate (mass flux) and the impact threshold (the smallest value of the wind shear velocity at which saltation can be sustained). We show that, in contrast to previous studies, the present model's predictions are in very good agreement with a range of experiments, as well as with numerical simulations of aeolian saltation. Because of its physical basis, we anticipate that our model will find application in studies of aeolian sand transport on both Earth and Mars

JLab Measurements of the 3He Form Factors at Large Momentum Transfers

The charge and magnetic form factors, FC and FM, of 3He have been extracted in the kinematic range 25 fm-2 < Q2 < 61 fm-2 from elastic electron scattering by detecting 3He recoil nuclei and electrons in coincidence with the High Resolution Spectrometers of the Hall A Facility at Jefferson Lab. The measurements are indicative of a second diffraction minimum for the magnetic form factor, which was predicted in the Q2 range of this experiment, and of a continuing diffractive structure for the charge form factor. The data are in qualitative agreement with theoretical calculations based on realistic interactions and accurate methods to solve the three-body nuclear problem

Polarization transfer in the d(epol,e' ppol)n reaction up to Q^2=1.61 (GeV/c)^2

The recoil proton polarization was measured in the d(epol,e' ppol)n reaction in Hall A of the Thomas Jefferson National Accelerator Facility (JLab). The electron kinematics were centered on the quasielastic peak (x_{Bj}~1) and included three values of the squared four-momentum transfer, Q^2=0.43, 1.00 and 1.61 (GeV/c)^2. For Q^2=0.43 and 1.61 (GeV/c)^2, the missing momentum, p_m, was centered at zero while for Q^2=1.00 (GeV/c)^2 two values of p_m were chosen: 0 and 174 MeV/c. At low p_m, the Q^2 dependence of the longitudinal polarization, P_z', is not well described by a state-of-the-art calculation. Further, at higher p_m, a 3.5 sigma discrepancy was observed in the transverse polarization, P_x'. Understanding the origin of these discrepancies is important in order to confidently extract the neutron electric form factor from the analogous d(epol,e' npol)p experiment.Comment: 6 pages, 4 figures; updated text, figures and table

JLab Measurement of the 4^4He Charge Form Factor at Large Momentum Transfers

The charge form factor of ^4He has been extracted in the range 29 fm$^{-2}$ $\le Q^2 \le 77$ fm$^{-2}$ from elastic electron scattering, detecting $^4$He nuclei and electrons in coincidence with the High Resolution Spectrometers of the Hall A Facility of Jefferson Lab. The results are in qualitative agreement with realistic meson-nucleon theoretical calculations. The data have uncovered a second diffraction minimum, which was predicted in the $Q^2$ range of this experiment, and rule out conclusively long-standing predictions of dimensional scaling of high-energy amplitudes using quark counting.Comment: 4 pages, 2 figure

A Search for Sigma^0_5, N^0_5 and Theta^++ Pentaquark States

A high-resolution (sigma_instr. = 1.5 MeV) search for narrow states (Gamma < 10 MeV) with masses of M_x approx 1500-1850 MeV in ep -> e'K^+ X, e'K^- X and e' pi^+ X electroproduction at small angles and low Q^2 was performed. These states would be candidate partner states of the reported Theta^+(1540) pentaquark. No statistically significant signal was observed in any of the channels at 90% C.L. Upper limits on forward production were determined to be between 0.7% and 4.2% of the Lambda(1520) production cross section, depending on the channel and the assumed mass and width of the state.Comment: 5 pages, 5 figures, to appear in Phys. Rev. C, update with responses to referee suggestion

Deeply Virtual Compton Scattering off the neutron

The present experiment exploits the interference between the Deeply Virtual Compton Scattering (DVCS) and the Bethe-Heitler processes to extract the imaginary part of DVCS amplitudes on the neutron and on the deuteron from the helicity-dependent D$({\vec e},e'\gamma)X$ cross section measured at $Q^2$=1.9 GeV$^2$ and $x_B$=0.36. We extract a linear combination of generalized parton distributions (GPDs) particularly sensitive to $E_q$, the least constrained GPD. A model dependent constraint on the contribution of the up and down quarks to the nucleon spin is deduced.Comment: Published in Phys. Rev. Let

Probing the high momentum component of the deuteron at high Q^2

The d(e,e'p) cross section at a momentum transfer of 3.5 (GeV/c)^2 was measured over a kinematical range that made it possible to study this reaction for a set of fixed missing momenta as a function of the neutron recoil angle theta_nq and to extract missing momentum distributions for fixed values of theta_nq up to 0.55 GeV/c. In the region of 35 (deg) <= theta_nq <= 45 (deg) recent calculations, which predict that final state interactions are small, agree reasonably well with the experimental data. Therefore these experimental reduced cross sections provide direct access to the high momentum component of the deuteron momentum distribution in exclusive deuteron electro-disintegration.Comment: 5 pages, 2 figure